On June 22, 2022 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported results from a retrospective, matched case control study (Press release, Aura Biosciences, JUN 22, 2022, View Source [SID1234616167]). This retrospective analysis assessed the visual acuity of patients following treatment with plaque radiotherapy compared with prospective data on visual acuity in subjects with early-stage choroidal melanoma treated with belzupacap sarotalocan by intravitreal administration in the Phase 1b/2 trial (NCT03052127).

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"These results point to the high unmet medical need for a first line vision preserving therapy for the treatment of early-stage choroidal melanoma given the high levels of irreversible visual acuity loss with the current standard of care with radiotherapy," said Carol Shields, MD, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University. "Being able to treat the disease early, avoid radiotherapy and spare long-term vision loss in many patients, as well as potentially reducing the risk of metastatic disease, could represent a paradigm shift in our approach to the treatment of choroidal melanoma. This would be a significant improvement in the quality of life for patients with this life-threatening rare disease."

Results from the Retrospective Study

Study Design

This retrospective, matched case control study compared visual acuity outcomes for 43 patients from Aura’s Phase 1b/2 trial evaluating intravitreal administration of belzupacap sarotalocan in patients with early-stage choroidal melanoma (AU-011-101, NCT03052127) to 150 patients from the subject database of a previously completed and published study where patients with small choroidal melanoma had been treated with plaque radiotherapy (Shields, et al. "Visual Outcome and Millimeter Incremental Risk of Metastasis in 1780 Patients With Small Choroidal Melanoma Managed by Plaque Radiotherapy." JAMA Ophthalmology. September 27, 2018). Both cohorts of patients were at high risk for vision loss due to having the tumor edge within 3.0 mm of the fovea. The patients were matched for tumor height, tumor diameter, distance from the fovea and baseline visual acuity, which are among the core factors that impact visual acuity after treatment.

Key Findings:

The vision results of patients with early-stage choroidal melanoma treated with radiotherapy showed the long term, progressive and irreversible loss of visual acuity in patients where tumors were close to the fovea.

The loss of vision in radiotherapy patients was ≥3 lines in a majority of patients as early as 2 years and ≥6 lines as early as 3 years.

We believe the comparison of the belzupacap sarotalocan and radiotherapy results supports the potential benefit of a targeted treatment achieving a statistically significant difference in visual acuity preservation as soon as two years including for both logMAR (Logarithm of the Minimum Angle of Resolution) vision (p = 0.0094) and change in logMAR vision (p = 0.0323).

We believe the progressive loss of visual acuity with radiotherapy observed in this retrospective study underscores the urgent need for a vision preserving targeted therapy.

The findings of this retrospective study were consistent with published clinical data supporting the irreversible loss of visual acuity after treatment with radiotherapy.

"We are committed to developing the first potential targeted therapy for patients with early-stage choroidal melanoma. We believe the visual acuity results of the retrospective matched case control study are exciting because they support the high unmet medical need for a long-term vision preserving therapy," said Dr. Cadmus Rich, Chief Medical Officer and Head of R&D of Aura Biosciences. "Belzupacap sarotalocan is currently being evaluated in a Phase 2 dose escalation clinical trial (AU-011-202, NCT04417530) using suprachoroidal administration in patients with early-stage choroidal melanoma. We remain on track to initiate our pivotal trial by the end of 2022."

Study Limitations include the retrospective nature and utilizing a matched case control design. The mean follow-up for patients treated with belzupacap sarotalocan in this initial analysis was 15.6 months. Due to the retrospective nature of this analysis, it is hypothesis-generating; no formal conclusions can be drawn. Aura has also initiated a prospective matched case control study to further evaluate the long-term visual acuity results of belzupacap sarotalocan from the Phase 2 trial AU-011-202 using suprachoroidal administration versus radiotherapy.

On June 22, 2022 Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported the signing of a definitive agreement for the sale of revenues from U.S. and European royalty and milestone interests in Klisyri (tirbanibulin) to Sagard Healthcare Partners and funds managed by Oaktree Capital Management, L.P. ("Oaktree") for $85 million (Press release, Athenex, JUN 22, 2022, View Source [SID1234616166]). Approximately $80 million of the proceeds from the transaction will be used toward partially paying down existing debt and operating the business, with $5 million to be placed into escrow and paid to Athenex upon the satisfaction of certain conditions. The transaction is subject to customary closing conditions.

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"We are executing on our new strategy and will continue to deliver on the objectives that we laid out to extend our cash runway by over 18 months. We are pleased to enter into this agreement, as we believe it generates meaningful benefits for our stockholders as we continue to pay down debt and extend our cash runway," said Dr. Johnson Lau, Chief Executive Officer of Athenex. "The sale of the revenues from the U.S. and European royalty and milestone interests in Klisyri represents another step in continuing to monetize non-core assets to focus on developing our potential best-in-class NKT cell platform."

Ladenburg Thalmann & Co. Inc. and Royalty/Revenue Interest Capital Advisors LLC served as financial advisors to Athenex and Cooley LLP served as legal counsel to Athenex. Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C. served as legal counsel to Sagard Holdings and Oaktree.

On June 22, 2022 Alpha Tau Medical Ltd. ("Alpha Tau"), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT, reported that the first patient was treated in a feasibility study evaluating the Alpha DaRT as a neoadjuvant therapy in patients with prostate cancer at the Rambam Health Care Center in Haifa, Israel (Press release, Alpha Tau Medical, JUN 22, 2022, View Source [SID1234616165]).

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The primary objectives of this study are to assess the feasibility and safety of intratumoral Alpha DaRT source implantation for the treatment of local prostate cancer prior to surgery. Secondary objectives include assessing radiological and pathological objective response rate of the tumor, and changes in quality of life measures. The study involves two separate procedures, with surgical resection of the prostate 50 days following Alpha DaRT source insertion, and can recruit up to 10 patients. The surgical resection will be performed at the Carmel Medical Center in Haifa, Israel using a da Vinci surgical robot, and will be led by Dr. Yuval Freifeld and his senior team.

"This is a historic day for Alpha Tau," said Alpha Tau CEO Uzi Sofer. "After gaining a wealth of experience in treating superficial cancers such as those of the skin, head & neck, and breast, we are thrilled to have treated the first prostate patient, our first internal organ treatment. Our pre-clinical data show that the Alpha DaRT technology may have potential to address a very broad range of solid tumor types, and our strategy is to expand our clinical studies across multiple tumor types in order to evaluate the full potential of the Alpha DaRT. This study suggests our ability to deliver this promising treatment beyond superficial tumors and is an important milestone in executing on this strategy."

Alpha Tau CMO Dr. Robert Den remarked, "Since the prostate is surgically removed 50 days after the Alpha DaRT insertion, the neoadjuvant design of this trial will allow us to analyze the pathological response of tissues to the Alpha DaRT. This will hopefully provide very valuable information to our researchers and allow us to compare theoretical versus actual cell damage."

Dr. Tomer Charas, head of the GU Radiotherapy Unit in the Oncology Division at Rambam Health Care Center in Haifa, Israel, and principal investigator of the study, commented, "We are excited to be leading this trial in collaboration with the Carmel Urology team, and to be the first to evaluate Alpha DaRT in prostate cancer in patients. Based on the results from preclinical studies, it is possible that neoadjuvant treatment with the Alpha DaRT may also trigger an immune response, which could help decrease the risk of tumor recurrence. In addition, a preferable safety profile has been observed to date in treatments of other tumor types."

On June 22, 2022 Plus Therapeutics, Inc. (Nasdaq: PSTV), a clinical-stage pharmaceutical company developing innovative, targeted radiotherapeutics for rare and difficult-to-treat cancers, and Biocept, Inc. (Nasdaq: BIOC), a leading provider of molecular diagnostic assays, products and services, reported a multi-year agreement to employ Biocept’s cerebrospinal fluid (CSF) assay CNSide1 in Plus Therapeutics’ ReSPECT-LM Phase 1/2a dose-escalation clinical trial of Rhenium-186 NanoLiposome (186RNL) for the treatment of patients with leptomeningeal metastases (LM), which is cancer in the membranes that surround the brain and spinal cord (Press release, Cytori Therapeutics, JUN 22, 2022, View Source [SID1234616164]).

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CNSide is an assay based on Biocept’s proprietary quantitative tumor cell capture method paired with advanced digital imaging and molecular markers used to detect, characterize and quantify tumor cells in CSF of patients with a variety of solid organ carcinomas and suspected LM, particularly breast and lung cancer which are leading causes of LM. CNSide provides a robust quantitative method to evaluate tumor status and response to treatment compared to conventional CSF cytology or radiologic monitoring.

"LM and the therapeutic response to 186RNL can theoretically be assessed through periodic sampling of tumor cells in the CSF," said Norman LaFrance, M.D., Chief Medical Officer and SVP of Plus Therapeutics. "Every LM patient in the ReSPECT-LM trial will have permanent access to the CSF via an intraventricular catheter placed in the cerebral ventricles before treatment, permitting medical staff to draw CSF as easily as blood. The CNSide technology is the most sophisticated and powerful technology available for monitoring tumor status and therapeutic response and can be seamlessly implemented into the ReSPECT-LM trial for the potential benefit of patients with LM."

"We see a significant opportunity for Plus Therapeutics to use our CNSide assay to monitor tumor burden in the CSF and response to treatment, and to profile specific cellular biomarkers which may inform their cancer radiotherapeutic drug development activities," said Michael Dugan, M.D., Biocept’s Chief Medical Officer and Medical Director. "CNSide has the potential to improve our understanding of therapy response in patients with LM treated with novel therapeutic approaches. This represents an area of very high unmet need in the care of cancer patients with certain forms of brain metastasis that are life-threatening."

On June 22, 2022 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), reported that the first patient has been dosed in the MM cohort of COVALENT-101, the company’s Phase I clinical trial evaluating BMF-219, Biomea’s covalent menin inhibitor, in patients with R/R AML, ALL, DLBCL, and MM (Press release, Biomea Fusion, JUN 22, 2022, View Source [SID1234616163]).

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"From the very beginning of our journey, we have been exploring and validating the broad potential of covalently inhibiting the scaffold protein, menin, in a host of liquid and solid tumors. Menin’s broad role in a variety of tumor types is rather striking. Based on the outstanding translational work of our team, we have seen compelling preclinical activity using BMF-219 in MM subtypes, especially those that are driven by MYC, a protein essential to the growth of numerous tumor types," said Steve Morris, MD, Chief Medical Officer of Biomea Fusion.

"Today, we have taken the first step to explore the clinical potential of BMF-219, a single-agent covalent menin inhibitor, in treating relapsed / refractory multiple myeloma patients. This represents the second cancer type, as well as the first cancer type outside of AML, to be studied with BMF-219. We are committed to delivering innovative medicine to patients in need, including those with R/R MM. I am incredibly proud of our team for their dedication as they continue to explore the boundaries of where science is leading us. Our mission is fundamentally driven by the wish to help cure patients of their disease. I would like to thank the entire Biomea team, including our participating clinical sites and wonderful collaborators, for their work quality and brilliance in achieving this very important milestone," said Thomas Butler, CEO, Chairman of the Board and Co-Founder of Biomea.

About COVALENT-101

A Phase I, open-label, multi-center, dose escalation and dose expansion study designed to assess the safety, tolerability, and pharmacokinetics / pharmacodynamics of once daily oral dosing of BMF-219 in patients with r/r acute leukemias —including subpopulations where menin inhibition is expected to provide a therapeutic benefit (e.g., patients with MLL1/KMT2A gene rearrangements or NPM1 mutations). The study has been expanded to include cohorts for patients with R/R multiple myeloma and R/R diffuse large B-cell lymphoma. Additional information about the Phase I clinical trial of BMF-219 can be found at ClinicalTrials.gov using the identifier NCT05153330.

About Multiple Myeloma (MM)

MM is a cancer of plasma cells, which make antibodies (immunoglobulins) and are mainly located in the bone marrow. As cancerous cells proliferate and migrate from the bone marrow, organ damage occurs due to excess immunoglobulins in bones and blood and the general weakening of bones. Approximately 35,000 people in the U.S. are diagnosed with MM each year and the 5-year relative survival rate is ~56% (Source: NCI SEER Data). The need is greatest among patients with relapsed or refractory disease, with overall survival as low as 6 months in some patients. Additionally, it is estimated that more than 60% of MM patients have menin dependent genetic drivers (MYC addicted or driven) and that these drivers are more common in the relapsed or refractory setting.